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Evaluating Luspatercept for Anemia in Adults and Adolescents with Alpha Thalassemia

A Phase 2, Study for the Treatment of Anemia With Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents

Phase 2 Interventional Bristol-Myers Squibb · NCT05664737

This study is testing if a new treatment called luspatercept can help adults and teens with alpha thalassemia feel better by improving their anemia symptoms compared to a placebo.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment189 (estimated)
Ages12 Years and up
SexAll
SponsorBristol-Myers Squibb Industry-sponsored
Locations36 sites (Halifax, Nova Scotia and 35 other locations)
Trial IDNCT05664737 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy and safety of luspatercept combined with best supportive care compared to a placebo with best supportive care in adults suffering from alpha-thalassemia hemoglobin H disease. It also seeks to evaluate the safety and pharmacokinetics of luspatercept in adolescent participants. The study will involve participants who are either transfusion-dependent or have not required transfusions for a specified period. The primary focus is on improving anemia symptoms and overall patient outcomes.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older or adolescents aged 12 to less than 18 with a documented diagnosis of alpha-thalassemia hemoglobin H disease and specific transfusion requirements.

Not a fit: Patients who do not have alpha-thalassemia or those who are not transfusion-dependent may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve anemia management and quality of life for patients with alpha-thalassemia.

How similar studies have performed: Other studies have shown promise in treating anemia with similar approaches, but the specific application of luspatercept in this context is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

* Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:.

  1. TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization.
  2. NTD participant:\< 6 RBC units during the 24 weeks prior to randomization(transfusion due to conditions other than A-Thal will not be considered)and, RBC transfusion-free during at least 8 weeks prior to randomization(unless transfusion was required to treat an acute medical condition other than A-Thal) and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded.
* Adult participant has Eastern Cooperative Oncology Group (ECOG) 34 score of 0 or 1.
* Adolescent participant 12 years to \< 18 years with documented diagnosis of A-Thal HbH disease with transfusion dependence defined as:.

  1. TD participant: ≥ 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for \> 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years.
  2. NTD participant:\< 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded.
  3. Participant has Karnofsky (age ≥16 years) or Lansky (age \< 16 years) performance status score ≥ 50 at screening.

Key Exclusion Criteria:

* Medical Conditions: Diagnosis of A-ThalTrait, Hb Bart hydrops, ATRx A-Thal, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Undergone episodes of hemolysis not related to A-Thal within the 8 weeks prior to randomization.
* Participant has deep vein thrombosis (DVT), stroke or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24weeks prior to randomization.
* Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment.
* Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding.
* Prior/Concomitant: Undergone HSCTs or gene therapy (candidates for HSCT or gene therapy with waiting period of ≥ 12 months are eligible).
* Use of hydroxyurea treatment ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants.
* Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to control the growth of EMH mass(es) during the screening period.
* Any medical or psychiatric condition (including active infections, recent surgery, sequelae of diseases or interventions, clinically significant laboratory abnormalities or concurrent treatment) that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data.
* Other protocol-defined inclusion/exclusion criteria apply.

Where this trial is running

Halifax, Nova Scotia and 35 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions AnemiaLuspaterceptBMS-986346ACE-536α-thalassemiaAlpha ThalassemiaReblozylTransfusions
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.